Denali Therapeutics Announces Plans to File for Accelerated Approval of Tividenofusp Alfa (DNL310) for the Treatment of MPS II

After recent successful meetings with the FDA, Denali Therapeutics announced its plan to file for accelerated approval of DNL310 for the treatment of MPSII, also known as Hunter Syndrome.

“We thank CDER for a positive and collaborative discussion and their guidance on CSF HS as a surrogate biomarker, which we see as a significant step towards accelerating development of medicines for individuals and families living with MPS II,” said Carole Ho, MD, Chief Medical Officer of Denali. “This milestone reflects a collective effort across the patient community, academia and industry to communicate the science and advocate for faster paths to effective treatments addressing these devastating rare diseases. We are excited by the potential to deliver a new MPS treatment sooner using the accelerated approval pathway. We also look forward to plans for conversion to full approval following completion of the global Phase 2/3 COMPASS study, and we are grateful for the continued participation and commitment of patients, clinicians, and study teams involved in the tividenofusp alfa clinical studies.”

Denali Therapeutics plans to submit biologics license application (BLA) early in 2025 under the accelerated approval pathway.

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